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Technology Review provides an update on genetic research involving Duchenne Muscular Dystrophy in dogs that provides hope for humans as well. Courtney Humphries writes about the new gene research and states,

An international team of researchers has successfully treated dogs
with the canine form of Duchenne muscular dystrophy (DMD), a rapidly
progressing and ultimately fatal muscle disease that afflicts one out
of every 3,600 boys. The researchers used a novel technique called exon
skipping to restore partial function to the gene involved in Duchenne.
The study, published in Annals of Neurology, gives hope that a similar approach could work in humans.

DMD is caused by an aberration in the gene that encodes dystrophin,
an important structural protein in muscle cells. Patients with DMD are
unable to produce functional dystrophin, which leads to holes in the
outer membranes of their muscle cells. Eventually, their muscles
degenerate faster than they can be rebuilt, and few patients survive
beyond their early 30s.

Unlike traditional gene therapy, which attempts to replace a mutated
gene with a functional copy, exon skipping relies on a variation of a
technique called antisense, in which short synthetic DNA or RNA
molecules are designed to bind to a region of DNA or RNA and block its
function. Companies are developing antisense therapies for cancer,
diabetes, heart disease, and autoimmune diseases, among others. . . .

Eric Hoffman,
a lead author of the study at Children's National Medical Center, in
Washington, DC, says that scientists realized they might help DMD
patients by creating a "patch" that blocks transcription of a portion
of the gene in a way that puts the remaining code back into
sequence--essentially recreating the milder Becker muscular dystrophy. . . .